(Q34193502)

31 July 2017

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title

Pricing and reimbursement of orphan drugs: the need for more transparency (English)

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31 July 2017

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Steven Simoens

1

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31 July 2017

publication date

17 June 2011

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Orphanet Journal of Rare Diseases

31 July 2017

published in

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31 July 2017

volume

6

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31 July 2017

page(s)

42

1 reference

https://scigraph.springernature.com/pub.10.1186/1750-1172-6-42

31 July 2017

exact match

0 references

cites work

Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: the Bosentan Patient Registry

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

10 years of NICE: still growing and still controversial

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Pharmaceutical risk-sharing agreements

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Exploring emerging technologies using metaphors--a study of orphan drugs and pharmacogenomics

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Choosing the right incentive strategy for research and development in neglected diseases

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Orphan drugs and the NHS: should we value rarity?

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Drugs for exceptionally rare diseases: do they deserve special status for funding?

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Endothelin antagonism in pulmonary hypertension, heart failure, and beyond

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

QALY maximisation and people's preferences: a methodological review of the literature.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

National Institute for Clinical Excellence and its value judgments

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Rare diseases, orphan drugs and their regulation: questions and misconceptions

5 July 2018

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-6-42

The Chinese healthcare system: structure, problems and challenges

21 January 2018

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-6-42

Prices and availability of biopharmaceuticals: an international comparison.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses?

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3132155

The problem of orphan drugs

28 October 2018

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-6-42

Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency

21 January 2018

1 reference

https://api.crossref.org/works/10.1186%2F1750-1172-6-42

Cost-effectiveness analysis and the consistency of decision making: evidence from pharmaceutical reimbursement in australia (1991 to 1996)

21 January 2018

1 reference

12 December 2020

Assessing the economic challenges posed by orphan drugs

1 reference

12 December 2020

Multiple sclerosis risk sharing scheme: a costly failure

1 reference

12 December 2020

Orphan drugs. Relating price determination to disease prevalence

1 reference

12 December 2020

The Orphan Drug Backlash

1 reference

12 December 2020

Identifiers

DOI

10.1186/1750-1172-6-42

1 reference

Dimensions Publication ID

31 July 2017

0 references

1 reference

31 July 2017

1 reference